Glycogen Storage DiseaseNCT00566878
Pompe Lactation Sub-Registry
An observational study of Glycogen Storage Disease and Pompe Disease, run by Genzyme, a Sanofi Company at 2 sites in United States.
Trial summary
The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with alglucosidase alfa and to measure breast milk production and composition in women with Pompe Disease who receive alglucosidase alfa. Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Glycogen Storage Disease, Pompe Disease
- What is being tested?
- alglucosidase alfa
- Type of treatment
- Injection or infusion
- How many people
- 2 participants
- Where
- 2 sitesUnited States
- Study dates
- 31 Mar 2012to 9 Feb 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Females diagnosed with Pompe Disease and Lactating
Who may be able to join
- Must be enrolled in Pompe Registry (NCT00231400)
- Must be pregnant and intend to breast-feed or be currently lactating and receive at least one infusion of alglucosidase alfa while lactating
- Provide a signed Patient Information and Authorization form to participate in the sub-registry prior to any sub-registry-related assessments are performed
- Agree to adhere to the sub-registry guidelines for antibody testing and recommended schedule of assessments.
Who cannot join
- Patients will be excluded from this sub-registry if they have received an investigational drug (excluding alglucosidase alfa in regions where alglucosidase alfa is not commercially available) within 30 days prior to Visit 1 breast milk collection
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Registry participation is worldwide. Facilities not yet active may enroll upon identification of a patient
Cambridge, Massachusetts, 02142
Study site
Grand Rapids, Michigan, 49503
About this study
Official title: A Sub-Registry to Determine the Presence of Alglucosidase Alfa in Breast Milk From Women With Pompe Disease Treated With Alglucosidase Alfa.
The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with alglucosidase alfa and to measure breast milk production and composition in women with Pompe Disease who receive alglucosidase alfa.
- Conditions
- Glycogen Storage Disease, Pompe Disease
- Being tested
- alglucosidase alfa
- Keywords
- Glycogen Storage Disease, GSD-II, Pompe Disease, Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis II
- Medical subject headings
- Glycogen Storage Disease, Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
alglucosidase alfaBiological
Also called Myozyme, Lumizyme
At least one infusion of alglucosidase alfa post partum
What is being measured
The primary outcome is the main question the study is designed to answer.
alglucosidase alfa accumulation in immature (1-3 months post partum) or mature (4-6 months post partum) breast milk sample from mothers with Pompe disease who are being treated with alglucosidase alfa.
Measured: 6 Months
breast milk production and composition in women with Pompe disease who receive alglucosidase alfa
Measured: 6 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2007 (estimated)
- Study start31 Mar 2012
- Primary completion9 Feb 2024
- Study completion9 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00566878
- Sponsor's protocol ID
- AGLU03406
- Other IDs
- LTS13972
- Status last verified
- Mar 2024
- First submitted
- 1 Dec 2007
- Last updated
- 19 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Sub-Registry to Determine the Presence of Alglucosidase Alfa in Breast Milk From Women With Pompe Disease Treated With Alglucosidase Alfa.
- ClinicalTrials.gov ID
- NCT00566878
- Acronym / sponsor ID
- AGLU03406
- Conditions
- Glycogen Storage Disease, Pompe Disease
- Keywords
- Glycogen Storage Disease, GSD-II, Pompe Disease, Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis II
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 31 Mar 2012 (actual)
- Primary completion
- 9 Feb 2024 (actual)
- Study completion
- 9 Feb 2024 (actual)
- First posted
- 4 Dec 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with alglucosidase alfa and to measure breast milk production and composition in women with Pompe Disease who receive alglucosidase alfa.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Females diagnosed with Pompe Disease and Lactating
Key inclusion criteria (4 listed)
- Must be enrolled in Pompe Registry (NCT00231400)
- Must be pregnant and intend to breast-feed or be currently lactating and receive at least one infusion of alglucosidase alfa while lactating
- Provide a signed Patient Information and Authorization form to participate in the sub-registry prior to any sub-registry-related assessments are performed
- Agree to adhere to the sub-registry guidelines for antibody testing and recommended schedule of assessments.
Key exclusion criteria (1 listed)
- Patients will be excluded from this sub-registry if they have received an investigational drug (excluding alglucosidase alfa in regions where alglucosidase alfa is not commercially available) within 30 days prior to Visit 1 breast milk collection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| alglucosidase alfaAlso: Myozyme, LumizymeAt least one infusion of alglucosidase alfa post partum | Biological | – |
Primary outcome
alglucosidase alfa accumulation in immature (1-3 months post partum) or mature (4-6 months post partum) breast milk sample from mothers with Pompe disease who are being treated with alglucosidase alfa.
Time frame: 6 Months
breast milk production and composition in women with Pompe disease who receive alglucosidase alfa
Time frame: 6 Months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease and Pompe Disease, run by Genzyme, a Sanofi Company at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
alglucosidase alfa accumulation in immature (1-3 months post partum) or mature (4-6 months post partum) breast milk sample from mothers with Pompe disease who are being treated with alglucosidase alfa. (time frame: 6 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2024.